Immunotherapy, mass-spectrometry peptide-based assays are paving the way for diagnostic and treatment revolution.

Precision medicine is on the cusp of transforming the diagnosis and management of many diseases, particularly cancer. From genetic sequencing to gene editing using CRISPR technology, laboratorians are on the leading edge of this revolution.

“For close to a century, many have dreamed of using the immune system to target cancer,” Milone told CLN Stat. “Over the past two decades, significant advances in our understanding of cancer and immunity, combined with the development of robust technologies to genetically manipulate immune cells, has led to the development of a number of immune-based therapies for cancer.”

“I believe we are on the precipice of bringing this exciting technology out of the research setting and into routine clinical practice of medicine,” added Milone.

The most mature of these approaches are antibody therapeutics such as Herceptin, he explained, which have transformed the treatment of certain cancers. Others include immune checkpoint inhibitors such as ipilimumab and pembrolizumab, which block CTLA-4 and PD-1, and genetically modified T cells that use viral vectors—like HIV-derived lentiviral vectors—to selectively target tumors.

“The technologies are evolving rapidly with new gene editing approaches now being applied,” Milone said. “Thus, the state of the art today in this area could very possibly be the past tomorrow.”

Milone’s talk focuses on adoptive cell immunotherapy using genetically engineered T cells, an approach that is furthest along in clinical development and may see regulatory approvals within the next year. His co-presenters plan to review gene therapy in hemophilia, obstacles in applying gene therapy to genetic disease and approaches to overcoming them, and advances in gene editing technology, including efforts to apply these approaches to blood diseases like sickle cell disease.

This is an important session for attendees, Milone said, given that gene therapies have already begun entering the clinical setting. “These therapies are certain to bring some new challenges to the lab medicine community,” he said.